Intravenous cyclophosphamide therapy for patients with severe ocular inflammatory diseases who failed other immunomodulatory therapies

Abstract

Purpose : This retrospective, observational study aimed to assess the safety and efficacy of cyclophosphamide (CP) therapy for patients with severe ocular inflammatory diseases who failed other immunomodulatory therapies (IMTs).

Methods : Medical records of 1295 patients who presented to Uveitis Clinic at Byers Eye Institute at Stanford between 2017-2022 were reviewed. Seven patients (10 eyes) who received CP therapy for ocular inflammatory diseases with >1 year of follow-up were included. Data review included demographics, ocular findings, previous and concomitant therapies, treatment outcomes, and adverse events.

Results : Mean age of patients (4 male, 3 female) was 61.6±14.9 (43.0-89.0) years. Clinical diagnoses included scleromalacia perforans (4 eyes, 3 patients), anterior necrotizing scleritis (1 eye, 1 patient), peripheral ulcerative keratitis (2 eyes, 1 patient), orbital pseudotumor (1 eye, 1 patient), HLA-B27 associated panuveitis and retinal vasculitis (2 eyes, 1 patient). Ocular disease was idiopathic in 3 patients, and was associated with rheumatoid arthritis, IgG-4 sclerosing disease, dermatomyositis, and ankylosing spondylitis in 1 patient each. All patients had history of previous IMT use including methotrexate (5 patients), mycophenolate mofetil (3 patients), azathioprine (1 patient), tacrolimus (1 patient), adalimumab (2 patients), infliximab (4 patients), and rituximab (1 patient). Mean follow-up time was 34.4±11.0 (13-45) months, and mean duration of CP therapy was 11.9±8.8 (5-28) months. Inflammation improved in all patients. Remission was achieved in 5 patients (71.4%). One patient achieved resolution of disease but continues CP while waiting for surgery, another patient had a flare of his scleritis and was restarted on CP leading to stabilization of disease. Four patients (57.1%) experienced transient leukopenia (WBC <4000/mL), one patient reported fatigue after infusions, one patient had single episode of fever, one patient reported a self-limited episode of abdominal pain.

Conclusions : CP therapy can be considered a potentially effective and relatively safe treatment option for patients with severe ocular inflammatory diseases who failed other IMTs including biologics (TNFa and CD20 inhibitors). As leukopenia was the most common side effect, close monitoring with regular blood work is essential in the utilization of CP for these patients.

This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.